HL2351 CAPS Phase II Study

Phase 2TerminatedNCT02853084

Handok Inc.8 enrolled

Overview

This is an open label, single arm trial to evaluate the efficacy, safety, and pharmacokinetics of HL2351 in patients with cryopyrin associated periodic syndromes (CAPS).

This trial enrolls patients with CAPS being on stable dose of anakinra daily and then switch over HL2351 given once weekly. It consists of 4 phases, 6-week screening, single dose PK, 6-month multiple dose, and 18-month extension phase.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Cryopyrin-Associated Periodic Syndromes (CAPS)

Interventions

HL2351DRUG

The dose will be adjusted based on disease activities

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: * Documented molecular diagnosis of NALP3 mutation or diagnosis of CAPS based on sign and symptom * Diagnosis of CAPS based on signs and symptoms * Current stable doses of anakinra, alone or in combination with methotrexate or corticosteroids Exclusion Criteria: * Patient been diagnosed with a hereditary periodic fever syndrome or autoinflammatory disease other than CAPS * Patients whose disease is inadequately controlled on current stable doses of anakinra, alone or in combination with methotrexate or corticosteroids

Locations (1)

Handok Inc.

Seoul, South Korea

Outcomes

Primary Outcomes

Change in Diary Symptom Sum Score (DSSS)

Time frame: from baseline to Month 6

Change in SAA, CRP, ESR levels

Time frame: from baseline to Month 6, from baseline to each visit up to Month 24

Change in physician's global assessment of autoinflammatory using 100mm VAS score disease

Time frame: from baseline to Month 6, from baseline to each visit up to Month 24

Change in patient's(parents or legal guardians) global assessment of autoinflammatory using 100mm VAS score disease

Time frame: from baseline to Month 6, from baseline to each visit up to Month 24

Pharmacokinetic profile(Cmax)